Most of these conditions aim at ensuring that germline genome editing will be used only to prevent a serious disease, where no reasonable alternatives exist, and under strong supervision. Some of them will be very difficult to meet. For instance, how can the long term follow-up of children (and their children) born with the help of genome editing be guaranteed? This would be specially difficult with people traveling to other countries to access these technologies.
Noncoding elements encompass more than 98% of the human genome and have been linked to regulatory sequences that contribute to human health and disease (1). Since the publication of the human genome sequence, considerable effort has been made to annotate functional elements, including noncoding regulatory regions--i.e., cisregulatory regions and noncoding RNAs (ncRNAs) that are involved in transcriptional regulation. Transcription factors often associate with hundreds to thousands of binding sites throughout the genome, and identifying which sites regulate gene expression often requires time-consuming and complex enhancer studies, or parallel assays in which short enhancer or promoter sequences are cloned into non-native contexts (2, 3). A recent study by Sanjana et al. (4) and a report by Fulco et al. (5) on page 769 of this issue address this obstacle using clustered regularly interspaced short palindromic repeats (CRISPR) screens to functionally characterize noncoding elements in their native context.
HIV-positive patients are often dependent on continuous antiretroviral therapy (ART) to prevent AIDS progression (such patients are called noncontrollers). A small proportion of individuals, called HIV posttreatment controllers, sustain HIV remission after receiving short-term ART. To understand the differences between these types of patients, Sharaf et al. sequenced HIV genomes from plasma samples. Before the interruption of ART, posttreatment controllers had a lower HIV genome reservoir size than noncontrollers by a factor of 7. Posttreatment controllers also had fewer defective HIV genome copy numbers and showed heightened HIV-specific immune responses and slower viral rebound after interruption of ART. Analysis of HIV genome characteristics could provide important information for designing an individual's optimal treatment plan.
OSAKA – Osaka University opened a research center for genome-editing technologies on Wednesday. It is the first university in Japan to set up a permanent research facility for such technologies. Genome editing involves technologies to insert and delete or replace DNA at a specific site in the genome of an organism or cell. Genome-editing technologies alter genes with greater accuracy than existing genetic-modification technologies and are coming into wider use. With 10 researchers, the Osaka University center will study efficient genome editing and analysis, and edit genes in rats and mice.
OSAKA – Osaka University has become the first university in Japan to set up a permanent research facility for genome-editing technologies following the opening of a new center on Wednesday. Genome editing involves technologies to insert and delete or replace DNA at a specific site in the genome of an organism or cell. The technologies alter genes with greater accuracy than existing genetic-modification technologies and are coming into wider use. Ten researchers at the Osaka University center will study efficient genome editing and analysis in rats and mice. It will also hold seminars for researchers from both Japan and abroad.